Blood cell therapy最新文献

{"title":"G-CSF-combined conditioning in allogeneic transplantation for non-remission acute myeloid leukemia with inv(3)(q21q26.2)/t(3;3)(q21;q26.2).","authors":"Yuki Oda, Seiko Kato, Maki Monna-Oiwa, Shohei Andoh, Yasuhito Nannya, Satoshi Takahashi, Takaaki Konuma","doi":"10.31547/bct-2024-035","DOIUrl":"10.31547/bct-2024-035","url":null,"abstract":"<p><p>Acute myeloid leukemia (AML) with inv(3)(q21q26.2) or t(3;3)(q21;q26.2) has a dismal prognosis and poor response to conventional chemotherapy. Allogeneic hematopoietic cell transplantation (HCT) is a potentially curative treatment for adult AML with inv(3)/t(3;3) during complete remission (CR). Nevertheless, because fewer than half of patients achieve a CR with induction conventional chemotherapy, allogeneic HCT is frequently performed for AML with inv(3)/t(3;3) in non-remission. Here, we report six patients with adult AML with inv(3)/t(3;3) in non-remission who underwent allogeneic HCT at our institute between 2010 and 2024. The median age at the time of HCT was 43.5 years (range, 28-53 years). The median proportion of blasts in the bone marrow at HCT was 47.5% (range, 0.7-75.0%). The median duration from diagnosis to HCT was 65.5 days (range, 41-123 days). A total of five patients received single-unit cord blood transplantation, and one received bone marrow transplantation from an HLA-matched sibling donor. All patients received a myeloablative conditioning regimen, including 12 Gy total body irradiation and granulocyte colony-stimulating factor (G-CSF) combined with high-dose cytarabine, as well as standard cyclosporine and methotrexate for graft-versus-host disease prophylaxis. With a median follow-up of 41 months for survivors, three patients experienced relapse at 18, 5, and 2 months, whereas the remaining three patients were alive and disease-free at 173, 110, and 30 months after HCT. Our data demonstrate that G-CSF-combined myeloablative conditioning following allogeneic HCT could lead to favorable long-term remission for adult AML with inv(3)/t(3;3) in non-remission at HCT.</p>","PeriodicalId":72423,"journal":{"name":"Blood cell therapy","volume":"8 1","pages":"181-185"},"PeriodicalIF":0.0,"publicationDate":"2025-02-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11883520/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143588478","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Haploidentical Hematopoietic Cell Transplantation in Dyskeratosis Congenita with Myelodysplastic Syndrome/Acute Myeloid Leukemia.","authors":"Sayan Sinha Roy, Manswinee Mallik, Alka Khadwal, Pradeep Kishore Reddy Gogulamudi, Arihant Jain, Gaurav Prakash, Pankaj Malhotra","doi":"10.31547/bct-2024-022","DOIUrl":"10.31547/bct-2024-022","url":null,"abstract":"<p><strong>Background: </strong>Allogeneic hematopoietic cell transplantation (Allo-HCT) is the only curative option for marrow failure, myelodysplastic syndrome (MDS), and acute myeloid leukemia associated with dyskeratosis congenita (DKC). Due to chromosomal instability and sensitivity to radiation and alkylating agents, HCT is associated with a high incidence of transplant-related mortality in DKC.</p><p><strong>Case report: </strong>A 25-year-old male presented with DKC-associated cutaneous manifestations and myelodysplastic syndrome / acute myelogenous leukemia (MDS/AML). Targeted next-generation sequencing revealed mutation of the DKC1 and RUNX1 genes. His mother and sibling sisters were carriers for the DKC1 mutation. Due to high donor-specific antibody mean fluorescence intensity (DSA-MFI) against the unshared Human Leukocyte Antigen-A (HLA-A) allele of his 6/12 HLA-matched father, his paternal cousin's sister was selected as a haploidentical (6/12 HLA-matched) donor for HCT. He underwent allo-HCT with stable disease burden using a specifically-designed RIC regimen containing treosulfan (at 50% reduced dosing), fludarabine, and rabbit anti-thymocyte globulin. The graft versus host disease (GVHD) prophylaxis contained reduced-dose post-transplant cyclophosphamide (PTCy dose reduction of 50%) with mycophenolate mofetil and cyclosporine. He engrafted with complete donor chimerism, and the day +30 marrow was in complete morphological remission with undetectable measurable residual disease by flow cytometry. On day +126, he developed steroid-responsive late-onset grade II acute GVHD (stage III skin GVHD). He suffered from morphologic relapse on day +220 and succumbed from sepsis with septic shock on day +256.</p><p><strong>Conclusion: </strong>This case demonstrates the safety and feasibility of haploidentical-HCT using a treosulfan-based reduced-intensity conditioning (RIC) regimen and modified PTCy-based GVHD prophylaxis in DKC. Disease relapse in this patient underscores the impact of pretransplant disease burden on relapse free survival in DKC patients with MDS/AML who are not eligible for myeloablative conditioning.</p>","PeriodicalId":72423,"journal":{"name":"Blood cell therapy","volume":"8 1","pages":"186-189"},"PeriodicalIF":0.0,"publicationDate":"2025-02-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11883479/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143588413","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Integrated Pediatric Intensive Care and Hematopoietic Stem Cell Transplantation Service Improves The Peri-Transplant Survival In Children.","authors":"Nalla Anuraag Reddy, Rachit Mehta, Indira Jayakumar, Anupama Nair, Vijayshree Muthukumar, Suresh Duraisamy, Venkateswaran Vellaichamy Swaminathan, Ramya Uppuluri, Revathi Raj","doi":"10.31547/bct-2024-014","DOIUrl":"10.31547/bct-2024-014","url":null,"abstract":"<p><strong>Background: </strong>Peri-transplant is a critical period which is associated with a myriad of complications that require pediatric intensive care unit (PICU) referral. PICU outcomes have been historically poor post-hematopoietic stem cell transplantation (HSCT), especially when associated with inotrope support, invasive ventilation, and renal replacement therapy. The study aimed to assess the outcomes of PICU referral in children undergoing HSCT.</p><p><strong>Patients & methods: </strong>A retrospective analysis was performed of children between 1 to 18 years of age who underwent HSCT between 2016 to 2023. A clinical deteriorating event (CDE) was defined as an unplanned transfer to the intensive care unit (ICU) or requiring ICU-level intervention on the floor. The reason for PICU referral, place of intervention, cause for the CDE, and requirement of respiratory, renal, and cardiac support were noted. The study period was divided into two 4-year intervals to assess change over time, 2016-2019 and 2020-2023.</p><p><strong>Results: </strong>In an eight-year period, a total of 934 HSCTs were performed, with 272 patients requiring PICU referral. A total of 415 CDEs were recorded. CDEs for PICU referrals were hypotension (43%), disproportionate tachycardia (42%), respiratory distress (26%), hypertension (22%), altered sensorium (8%), seizures (7.4%), and major bleeds (7.3%). Overall peri-transplant survival was 73.8% (n=201/272). Comparing the two study intervals, 2016-2019 and 2020-2023, the survival of patients on mechanical ventilation had improved from 4.5% to 27.5% (<i>p</i>=0.005) and from 39.4% to 55.9% (<i>p</i>=0.11) among those who received inotropes. Patients with three organ dysfunctions had worse outcomes. Disproportionate tachycardia [OR 0.19 CI 95% (0.06-0.64); <i>p</i>=0.008], hypotension [OR 0.177 CI 95% (0.04-0.84); <i>p</i>=0.029] and acute GVHD [OR 28.46 CI 95% (3.66-221); <i>p</i>=0.001] were significant risk factors for peri-transplant mortality as per multivariate analysis.</p><p><strong>Conclusion: </strong>Integrated care with the PICU team is the first step towards improving survival in these critically ill children. With timely intervention on the floors for CDEs and protocol-driven care in the PICU, we have demonstrated an increase in overall survival over the past four years and would recommend similar team-based care for units catering to children.</p>","PeriodicalId":72423,"journal":{"name":"Blood cell therapy","volume":"8 1","pages":"173-180"},"PeriodicalIF":0.0,"publicationDate":"2025-02-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11883477/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143588481","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Fecal Microbiota Transplantation for Refractory <i>Clostridioides difficile</i> Infection Post Haploidentical Transplant for Pediatric Acute Myeloid Leukemia.","authors":"Pronamee Borah, Vipul Gautam, Vikram Kumar, Bhaskar Saikia, Rahul Naithani","doi":"10.31547/bct-2024-015","DOIUrl":"10.31547/bct-2024-015","url":null,"abstract":"<p><strong>Background: </strong><i>Clostridioides difficile</i> (<i>C. difficile</i>) infections are common in immunosuppressed patients. Sometimes these are difficult to treat in post-bone marrow transplant situations.</p><p><strong>Methods: </strong>A 2-year-old child with relapsed acute myeloid leukemia underwent a haploidentical bone marrow transplant. He developed 30-40 episodes/day of loose watery stools on day +19. The stool was positive for <i>C. difficile</i> infection. He failed oral vancomycin and metronidazole therapy. He received a fecal microbiota transplant (FMT) on day +43. The donor was the same sister who donated hematopoietic stem cells.</p><p><strong>Results: </strong>Three days later (day +46), stool frequency reduced from 22-24/day to 12-14/day. Color normalized to yellow and consistency improved from watery to semisolid without blood. He was discharged from the hospital 10 days after FMT on oral vancomycin and nasogastric feeding. Stool tested for <i>C. difficile</i> 16 days after FMT was negative and oral vancomycin was stopped.</p><p><strong>Conclusion: </strong>Fecal microbiota transplant could be a useful modality in children with severe <i>C. difficile</i> infection post-bone marrow transplant.</p>","PeriodicalId":72423,"journal":{"name":"Blood cell therapy","volume":"8 1","pages":"170-172"},"PeriodicalIF":0.0,"publicationDate":"2025-02-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11883475/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143588474","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"High Risk of Graft Rejection in Patients with HLA Homozygosity Following Haploidentical Transplant Using Post-Transplant Cyclophosphamide.","authors":"Nishant Jindal, Kaumil Patel, Malini Garg, Sumeet Mirgh, Akanksha Chichra, Lingaraj Nayak, Anant Gokarn, Sachin Punatar, Bhausaheb Bagal, Libin Jacob Mathew, Selma D'Silva, Meenakshi Singh, Navin Khattry","doi":"10.31547/bct-2024-024","DOIUrl":"10.31547/bct-2024-024","url":null,"abstract":"<p><p>Graft rejection is an important concern following haploidentical transplant with a reported incidence of 10-15%. The number of human leukocyte antigen (HLA) mismatches and the vector of mismatch have not been found to be associated with the risk of graft rejection in haploidentical transplants. Patients with HLA homozygosity at all loci (HLA-A, B, C, DRB1, and DQB1) undergoing haploidentical transplant is a rare scenario that results in zero mismatches in the graft-versus-host (GvH) direction and 2-5 mismatches in the host-versus-graft (HvG) direction depending on the donor haplotype. This results in a heavily skewed vector of HLA mismatch with unopposed allo-reactivity in the HvG direction. We reviewed our haploidentical transplant database for patients who were homozygous at all five loci and studied their outcomes. Seventy-one patients underwent haploidentical transplant at our center for malignant indications between July 1, 2010, and June 30, 2022. All but one patient received PTCy-based graft-versus-host disease (GvHD) prophylaxis. Of these 71 patients, two were homozygous at all five loci, and both patients developed graft rejection (100%). This was significantly higher than the risk of rejection in the remaining 69 patients where 5/69 (7.2%) had rejection (<i>p</i>=0.0085). Herein, we describe these two cases and review the literature on the impact of patient HLA homozygosity on graft rejection in patients undergoing haploidentical transplant.</p>","PeriodicalId":72423,"journal":{"name":"Blood cell therapy","volume":"8 1","pages":"167-169"},"PeriodicalIF":0.0,"publicationDate":"2025-02-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11883474/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143588417","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Eltrombopag Enhances Recovery from Cytopenias Due to Poor Graft Function after Hematopoietic Cell Transplantation.","authors":"Khalid Halahleh, Maram Al-Ya'Goub, Mohamad Ma'koseh, Rozan Al-Far, Waleed Da'na, Rula Al-Najjar Pharm, Ayat Taqash, Isra Muradi, Maher Sughayer, Husam Abu-Jazar","doi":"10.31547/bct-2024-017","DOIUrl":"10.31547/bct-2024-017","url":null,"abstract":"<p><strong>Introduction: </strong>Poor graft function (PrGF) is a serious complication of allogeneic hematopoietic cell transplantation (allo-HCT), and current therapies are only partially effective. Eight published reports on the effect of eltrombopag on PrGF showed encouraging results. This study aimed to assess the safety and efficacy of eltrombopag for the treatment of PrGF after allo-HCT.</p><p><strong>Methods: </strong>A retrospective study was conducted on 23 patients with PrGF following allo-HCT at the King Hussein Cancer Center (KHCC), Amman, Jordan between January 2013 and December 2023. The patient-, disease-, and transplant-related characteristics were obtained from the KHCC-BMT database.</p><p><strong>Results: </strong>The median age was 47 years (range, 26-68 years), and 13 patients were female. Fourteen patients received reduced-intensity conditioning, and 14 donors were human-leukocyte antigen (HLA) -identical siblings. Median hemoglobin (Hb) concentration pre-eltrombopag administration was 8.5 g/dL (range: 6-14.2 g/dL), granulocytes 7.1 × 10⁹/L (range: 1.6-85 × 10⁹/L), and platelets 18 × 10⁹/L (range: 6-50 × 10⁹/L). Thirteen patients had platelets < 20 × 10⁹/L, and 15 had reduced megakaryocytes. The median CD34-positive cell dose was 6.10 × 10⁶/kg (range: 2.75-9.78 × 10⁶/kg). Eltrombopag was initiated at a median of 105 day (d) post-transplant (range: 29-800 d). The median weekly dose was 488 mg (range: 350-700 mg), and the median treatment duration was 75 d (range: 5-446 d). In total, 65% of patients (n=15) responded at a median of 30 d (range, 7-122 d). Twelve responders were alive at a median follow-up of 30 months (range: 7.0-122 months) with normal blood counts. The 2-year overall rate was 48% (95% confidence intervals, 27-70%), and seven non-responders died because of relapse. No major adverse events were reported.</p><p><strong>Conclusion: </strong>Eltrombopag improved peripheral blood counts in patients with PrGF following allo-HCT. Thus, response to eltrombopag was predictable and has a significant effect on survival.</p>","PeriodicalId":72423,"journal":{"name":"Blood cell therapy","volume":"8 1","pages":"160-166"},"PeriodicalIF":0.0,"publicationDate":"2025-01-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11883521/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143588466","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Outcomes in patients undergoing hematopoietic stem cell transplantation for myelodysplastic syndromes.","authors":"Sujith Karumathil, Uday Kulkarni, Sushil Selvarajan, Sharon Lionel, Anup J Devasia, Fouzia N Aboobacker, Kavitha M Lakshmi, Anu Korula, Alok Srivastava, Aby Abraham, Vikram Mathews, Biju George","doi":"10.31547/bct-2024-002","DOIUrl":"10.31547/bct-2024-002","url":null,"abstract":"<p><p>Hematopoietic stem cell transplantation [HSCT] is the only curative option for patients with myelodysplastic syndromes [MDS]. Between 1991 and 2021, 154 patients [high risk, 86; low risk, 68] including 22 children underwent HSCT with a median age of 36 years. Conditioning regimens were myeloablative [n=97] and reduced intensity [n=53]. Donors were human leucocyte antigen (HLA)-matched related donors (MRDs) in 113 and alternate donors in 41. The graft source was peripheral blood stem cells in 92%. Engraftment occurred in 126 [81.9%] at a median of 15 days while 20 [12.9%] died before engraftment and eight [5.2%] had primary graft failure. Sinusoidal obstruction syndrome was seen in 27 [17.5%]. Grade 2-4 acute graft versus host disease [GVHD] occurred in 46.3% while Grade 3-4 GVHD was seen in 34.9% and the incidence of chronic GVHD was 69.4%. Bacterial infections occurred in 38 (24.6%) while viral infections were seen in 31 [20.1%], mainly cytomegalovirus, and invasive fungal disease in 17.5%. At a median of 33 months, 65 patients were alive; 14 (9.1%) had disease relapse, and 10 (6.5%) had secondary graft failure. The five-year overall survival (OS) (time from allogenic HSCT to death due to any cause) and event-free survival (time from allogenic HSCT to relapse/ progression of disease or death) were 41.69±4.2% and 40.8±4.4%, respectively. The five-year OS was significantly better in children [71%]. Outcomes were better with MRDs [45%] compared to alternate donors [29%; <i>p</i>=0.035]. Outcomes of HLA-MRD transplants have been improving; 44% for 1990 - 2000, 35% for 2001 - 2010, and 51% for 2011 - 2021. On multivariate analysis, age (adolescents and young adults [hazard ratio (HR) 2.7, <i>p</i>=0.021] and older adult age group [HR 3.6, <i>p</i>=0.006]), minor blood group mismatch [HR 2.0, <i>p</i>=0.028], bidirectional blood group mismatch [HR 2.6, <i>p</i>=0.010], and haploidentical stem cell donor [HR 2.2, <i>p</i>=0.007] were associated with poorer OS. In conclusion, outcomes of HSCT for MDS are reasonable among matched sibling donors but outcomes in alternate donors require improvement. Strategies to reduce GVHD and infections should be explored.</p>","PeriodicalId":72423,"journal":{"name":"Blood cell therapy","volume":"8 1","pages":"147-159"},"PeriodicalIF":0.0,"publicationDate":"2025-01-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11883483/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143588492","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"ROLE OF AUTOLOGOUS STEM CELL TRANSPLANTATION IN RELAPSED/ REFRACTORY HODGKIN'S LYMPHOMA: RETROSPECTIVE DATA FROM A TERTIARY CARE CENTRE IN INDIA.","authors":"Mangai Suseela Murugesan, Jayachandran Perumal Kalaiyarasi, Nikita Mehra, Parathan Karunakaran, Venkatraman Radhakrishnan, Krishnarathinam Kannan","doi":"10.31547/bct-2024-011","DOIUrl":"10.31547/bct-2024-011","url":null,"abstract":"<p><p>Hodgkin Lymphoma is a highly curable malignancy, and relapsed/refractory Hodgkin lymphoma (RRHL) is treated with salvage chemotherapy, followed by autologous stem cell transplant (ASCT). This single-center, retrospective study included patients with RRHL who underwent ASCT. The study included 62 patients with a median age at diagnosis of 22.5 years and a median age at transplant of 24.5 years. Advanced disease at presentation was observed in 64% of the patients. At relapse, 33.3%, 36.6%, and 30% had stage 2, 3, and 4 disease, respectively. Multiple salvage regimens were administered as per the practices at our institution. Among the patients, 63% had complete remission prior to transplant, and 74% underwent ASCT after first-line salvage. Different conditioning regimens were chosen according to institutional practice. Febrile neutropenia was observed in 93.5% of the patients. The treatment-related mortality rate was 4.8% (n=3). The median follow-up duration was 30 months. Median relapse-free survival (RFS) and overall survival (OS) were not reached, and the 3-year RFS and OS rates were 67.1% and 78%, respectively. Factors that adversely influenced OS and RFS were the type of last salvage used and the number of salvage lines prior to transplantation. An advanced stage at the diagnosis was associated with inferior OS but not with RFS. Patients with the refractory disease had inferior RFS and OS rates, of 25.2% and 56.6%, compared with patients with chemoresponsive disease, who had RFS and OS rates of 77% and 84.7%, respectively. ASCT in RRHL showed promising survival, with over two-thirds of the patients surviving without relapse after 3 years.</p>","PeriodicalId":72423,"journal":{"name":"Blood cell therapy","volume":"8 1","pages":"138-146"},"PeriodicalIF":0.0,"publicationDate":"2024-12-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11883480/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143588493","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Sirolimus as add-on therapy to corticosteroids for moderate-severe chronic graft versus host disease.","authors":"Ritika Sharma, Andrew Pardeep, Renaissa De, Shaweta Kaundal, Amol N Patil, Charanpreet Singh, Aditya Jandial, Arihant Jain, Gaurav Prakash, Alka Khadwal, Pankaj Malhotra, Deepesh P Lad","doi":"10.31547/bct-2024-016","DOIUrl":"10.31547/bct-2024-016","url":null,"abstract":"<p><strong>Introduction: </strong>The first-line treatment of moderate-severe chronic graft versus host disease (cGVHD) involves systemic corticosteroids ± calcineurin inhibitors. Around half of the patients will need second-line agents for corticosteroid-refractory/dependent (SR/SD) cGVHD. Herein, we report our experience using sirolimus as an add-on agent to corticosteroids in moderate-severe cGVHD.</p><p><strong>Methods: </strong>This was a single-center study of allogeneic cell transplant recipients aged ≥ 12 during 2016-2022. The diagnosis and severity of cGVHD were as per the NIH-2014 criteria. At the physician's discretion, sirolimus was added to corticosteroids for moderate to severe cGVHD. The GVHD response was classified based on the EBMT-NIH-CIBMTR criteria.</p><p><strong>Results: </strong>cGVHD occurred in 66 (49%) out of 134 recipients. It was mild in 13 (10%) and moderate-severe in 53 (39%) recipients. Sirolimus was used in 38 out of 53 (71.6%) patients with moderate-severe cGVHD, with equal proportions of matched-related (53%) and haploidentical HCT (47%) recipients. The median time to onset of cGVHD was 140 days (IQR 108-182). The onset was de novo in 14 (37%), quiescent in 15 (39%), and progressive in 9 (24%) patients. The median duration on sirolimus was 283 days (134-640). cGVHD was controlled in 30 (79%) and active in 8 (21%) recipients at 6 months. Dyslipidemia was the most common (73%) adverse event. Failure-free survival at two years was 61% (95% CI 38-78%).</p><p><strong>Discussion: </strong>This study demonstrates the safety and efficacy of sirolimus as an add-on agent to systemic corticosteroids in managing moderate-severe cGVHD. This strategy can reduce the burden of SR/SD cGVHD.</p>","PeriodicalId":72423,"journal":{"name":"Blood cell therapy","volume":"7 4","pages":"124-128"},"PeriodicalIF":0.0,"publicationDate":"2024-11-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11620984/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142803663","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Impact of the COVID-19 outbreak on the field of hematopoietic cell transplantation in the Asia-Pacific region: APBMT Activity Survey 2020/2021.","authors":"Minako Iida, Aloysius Ho, Xiao Jun Huang, Kaiyan Liu, Meng Lv, He Huang, Yachiyo Kuwatsuka, Joon Ho Moon, Yoon Seok Choi, Jong Wook Lee, Navin Khattry, Kavitha M Lakshmi, Leonie Wilcox, Amir Ali Hamidieh, Maryam Behfar, Bor-Sheng Ko, Kim Wah Ho, Gin Gin Gan, Udomsak Bunworasate, Suradej Hongeng, Usanarat Anurathapan, Tasneem Farzana, Joycelyn Sim, Vincent Lee, Man Kit Garret Leung, Phu Chi Dung, Van Man Huynh, Wasantha Rathnayake, Prasad Abeysinghe, Marjorie Rose Bravo, Teresita Dumagay, Aye Aye Gyi, Damai Santosa, Desta Ardini, Bishesh Sharma Poudyal, Mafruha Akter, Khishigjargal Batshkh, Mikhail Drokov, Chi-Cheng Li, Shinichiro Okamoto, Alok Srivastava, Anthony Dodds, Yoshiko Atsuta","doi":"10.31547/bct-2024-020","DOIUrl":"10.31547/bct-2024-020","url":null,"abstract":"<p><p>COVID-19 became a global pandemic in 2020 and significantly affected the activity of hematopoietic cell transplants (HCT) worldwide. Despite these challenges, a total of 28,793 transplants, including 18,518 allogeneic and 10,275 autologous transplants, were performed in 719 facilities in 2020 in the Asia-Pacific (AP) region. This represented a 5.1% increase in allogeneic transplants and a 3.1% increase in autologous transplants, an overall increase of 4.4% compared to the numbers in 2019. With respect to the donor source, haploidentical transplants increased significantly by 18.6%, related transplants by 8.8%, and cord blood transplants (CBT) by 9.2%. However, the number of unrelated transplants, excluding CBT, decreased for the first time by 8.2%. As a result, COVID-19 facilitated the growth of haploidentical transplants due to cross-border restrictions. Regarding the changes in the total number of transplants by country/region in 2020, it increased by 2,048 transplants in China, followed by Japan (210 transplants) and Korea (230 transplants); however, 14 of the 22 countries and regions decreased their number of transplants in 2020 compared to the previous year. There was no correlation between the increase or decrease in the number of transplants in 2020 and the Gross National Income (GNI) per capita of each country/region in 2020, as well as Domestic General Government Health Expenditure as a percentage of General Government Expenditure (GGHE-D/GGE). In 2021, the total number of transplants in this region was 34,754. With the exception of a few countries/regions that decreased the number of transplants in 2020, most countries/regions have started to see a recovery in 2021. The COVID-19 pandemic significantly affected the supply chain and logistics involved in HCT rather than its numbers; however, we have found ways to overcome logistical challenges to carry out transplant medicine without delay, even under these circumstances.</p>","PeriodicalId":72423,"journal":{"name":"Blood cell therapy","volume":"7 4","pages":"129-137"},"PeriodicalIF":0.0,"publicationDate":"2024-11-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11620985/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142803651","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}